Developing Gene Therapy For DMD

Our Partnership

We are co-funding a project called 'Developing gene therapy for Duchenne muscular dystrophy' by Professor George Dickson, of Royal Holloway University, London.

What Is Transplicing?

This gene therapy project aims to deliver a functioning full size dystrophn gene to a muscle cell, using a harmless virus. Previous research has delivered a less than full size gene (a mini- or micro-dystrophin gene) because the information in the dystrophin gene was too large for one virus to carry.

This approach tackles this problem by using two or three viruses, each carrying a different part of the dystrophin gene. When in the muscle cell, the different parts join together to form a full size dystrophin protein. This approach is called transplicing

What Is Happening With Clinical Trials?

This research aims to use gene therapy to create the full size dystrophin protein. If this is successful this could lead to further pre-clinical work which could lead to clinical trials.

The project was funded through the Duchenne Forum. The other members of the Duchenne Forum are MDUK, The Duchenne Research Fund, Harrison's Fund and Alex's Wish.

Most read

Duchenne affects approximately 1 in every 3,500 boys that are born but only around 1 in every 50 million girls. It may be rare, but it does happen. We have been speaking to Feriel, a 26 year old woman living with Duchenne muscular dystrophy. She has written us a short blog about her experiences with Duchenne from diagnosis to now.

Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

We are proud to share with you our first ever Impact Report, read about the very real impact we are having and the many things we have been working on over the past six years to end Duchenne.
Read more

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